The Voices

Rachel Smalley

Spinal Muscular Atrophy (SMA)

If there is a disease that illustrates the overwhelming failure of New Zealand’s antiquated approach to modern medicines, it is Spinal Muscular Atrophy (SMA).

Rachel Smalley has been travelling through New Zealand meeting families caught up in our unfunded medicines crisis.

SMA is the biggest genetic killer of New Zealand children under the age of two. Some children, with less severe forms of SMA, will never walk. Others will suffer challenging respiratory issues or scoliosis, or are fed through a tube after losing the ability to chew or swallow. Their parents, left grappling with an ever-evolving list of complex health issues, often struggle to see even the dimmest of lights on the horizon.

The greatest tragedy in all of this is that SMA is treatable. This suffering – and it is acute suffering – is avoidable. Some 58 countries in the developed world fund at least one drug to treat SMA. New Zealand is an outlier. Here, we have metaphorically rammed our fingers in our ears, stepped to one side, and watched our children die or waste away from this crippling disease. New Zealand doesn’t treat SMA.

What’s the issue? Money. Pharmac, our drug-buying agency, is under frequent attack for its failure to fund some of the life-transforming drugs widely available in so many of our peer nations. But Pharmac can’t fix this crisis. The issue has become a behemoth. Years and years of chronic underfunding mean Pharmac is now playing God, robbing money from one disease to treat another, shuffling a few million from here to there, and ultimately being forced to choose who will live and who will die. This crisis is not Pharmac’s to solve. It is the Government’s. The decision is one of funding, and ultimately lies with Jacinda Ardern.

In the 1990s, Pharmac was created with a mandate to introduce price competition to New Zealand’s pharmaceutical market, and stop Big Pharma rorting the system. It worked. The new monopolistic model did a great job of bulk-buying the mass-use drugs of the 1990s which allowed the Government to subsidise medicines or provide them for free.

Fast forward to 2021, more than a quarter of a century later, and no Government has revisited Pharmac’s objectives. It is mandated to operate in a silo and its key priority remains locked in – save money.

Not surprisingly, given the rapid developments in medical science, New Zealanders now find themselves in a dire situation; we spend less on medicines per capita, wait years to buy new drugs, and we languish at the very bottom of the developed world for access to modern medicine. Sit with that for a moment, New Zealand. We’re in last place.

Spinal Muscular Atrophy has, reluctantly, become the Poster Child of New Zealand’s medicines crisis. In recent years, three breakthrough treatments – Spinraza, Risdiplam and Zolgensma – have been developed to treat SMA. Spinraza, the most high-profile of the three, is publicly funded in 58 countries, including Australia.

In 2018, New Zealand and Australia both received funding applications for Spinraza. That same year, Australia’s SMA families were granted access to the life-saving drug. In New Zealand, three years later, Spinraza sits on a waiting list. It’s one of almost 100 modern medicines still waiting for funding.

Is it expensive? Yes. Like most modern medicines, Spinraza has been in development for years and drug companies, operating in a capitalist world, always look for a return on their $1b-$2b development cost. Several industry sources say the cost will be somewhere between $6m-$10m to treat around 35 children. In a modern, developed, wealthy nation, we need to ask ourselves if that is an excessive or acceptable price to prevent child suffering and infant mortality.

The Spinraza predicament also reveals an alarming absence of cost-benefit analysis within Pharmac and the Ministry of Health. There is no requirement to financially model the cost to society of not funding the drug.  Pharmac does not consider an SMA patient’s all-of-life dependency on our health system, the loss of parental family income, the impact on mental health, the cost of retro-fitting the family home to make it wheelchair-accessible, or the need for additional in-classroom support. It is an embarrassingly primitive approach which, undoubtedly, costs the taxpayer dearly.

If you can’t find it in yourself to accept the humanitarian case to treat SMA, you must surely agree it makes financial sense.

Early diagnosis is vital with SMA, but New Zealand doesn’t include the disease in the Newborn Screening Programme. Infants are screened for 23 genetic conditions soon after birth, but despite being the biggest genetic killer of under-2s in New Zealand, SMA is not included in the programme.

I asked a neurologist if it was a cost issue. Apparently not. The cost of adding SMA to the programme is miniscule. The reason we don’t screen for SMA is distressingly cynical. If we don’t treat a disease, we don’t screen for it.

On its website, the Newborn Screening Programme speaks to the importance of its work saying “…. the benefits of screening are enormous”. It says early diagnosis and treatment can improve the health of babies, prevent severe disability and even death. Just not if your baby has SMA.

It’s only when a child begins to deteriorate that our health system steps in and starts to investigate. Even with a diagnosis, all a neurologist can do in New Zealand is monitor the child’s decline. Some families have caught the next plane to Brisbane, choosing to leave New Zealand and live in Australia as medical refugees. Anything to access Spinraza for their child.

Finally, SMA also highlights the Ministry of Health’s entrenched pedestrian and reactionary approach to new technologies. It beggar’s belief, but the Ministry does not currently have a Medicines Strategy. There has been no thinking, no planning, no horizon-scanning, and no preparedness for the highly engineered, DNA-specific medications and gene therapies that are steaming towards us. Drugs like Spinraza and Risdiplam are just two treatments in an avalanche of life-transforming drugs that New Zealanders need access to. And yet, we haven’t given it a single thought.

Pharmac issued a statement earlier this year indicating it needed $417m to address over 70 medicines still waiting for funding. It got $200m spread over four years. Just $40m was set aside for this year.

Our multi-billion-dollar taxpayer response to the COVID-19 virus has taught us all to consider our own mortality, but for thousands of ill New Zealanders, they were doing that long before the virus arrived on these shores.

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Achieve a measurable, political commitment to reform Pharmac and create a fit-for-purpose drug-buying agency that supports and enables greatly improved access to modern medicines – and ensure a direct line of political accountability.




Introduce a globally accepted modern, cost-benefit analysis for medicines and medical devices which looks at the ‘value’ of a medicine, and considers the financial, economic, and social impact of untreated disease on our society.




Develop a Medicines Strategy to guide the decision-making process, create measurable targets to reduce Pharmac’s waiting list, and detail how the agency will respond to rapid developments in modern medicine to improve health outcomes for New Zealanders.

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